Evaluating the safety of midostaurin and chemotherapy in acute myeloid leukemia with abnormal genes

This study aimed to further investigate the safety and tolerability of midostaurin plus chemotherapy in patients with newly diagnosed FLT3 mutation-positive acute myeloid leukemia.  

This study concluded that this combination was safe and effective in these patients. 

FLT3 is a mutation (gene change) in leukemia cells. This mutation encourages the growth of leukemia cells. Therefore, FLT3 mutation-positive AML is an aggressive form of leukemia that is likely to come back after treatment. Newer targeted treatments are needed for these patients.

Midostaurin (Rydapt) is a targeted therapy for acute myeloid leukemia (AML). It works by blocking the action of an abnormal protein found on cancer cells. This stops the spread of AML. The combination of midostaurin and chemotherapy for the treatment of adults with newly diagnosed FLT3 mutation-positive AML was approved based on a previous study. However, the safety and tolerability of midostaurin plus chemotherapy needed to be further investigated. 

This study involved 103 patients with newly diagnosed FLT3 mutation-positive AML. Patients received midostaurin with induction chemotherapy such as cytarabine (Cytosar-U) with daunorubicin (Cerubidine) or idarubicin (Idamycin) and high-dose cytarabine consolidation chemotherapy. Induction treatment is the first treatment meant to cure a disease. Consolidation treatment is meant to keep remission as long as possible and prevent the cancer from coming back. 

Overall, 99% of patients reported at least one side effect during either phase of treatment. The most common side effects were low levels of white blood cells with fever, nausea, and diarrhea. Serious side effects occurred in 50% of patients. No new safety events were observed. 

74% of the patients who received induction treatment were in complete remission. Overall, 51% of patients went on to receive a stem cell transplantation.

This study concluded that midostaurin was safe in association with chemotherapy and was associated with low on-treatment relapse rates for newly diagnosed FLT3 mutation-positive AML.  

This study had a small number of participants and a short follow-up period. Novartis, the manufacturer of midostaurin, funded this study.